While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...

Donald Trump continues to make waves in biopharma; Sage rejects Biogen’s unsolicited takeover offer; the obesity space sees ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...

Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...

Joshua Mercieca had a rare condition called Duchenne muscular dystrophy, which is a genetic disorder characterised by ...

The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes ...

Roche has reported positive topline outcomes from the second year of the multinational Phase III EMBARK trial of Elevidys ...

Sarepta Therapeutics (NASDAQ:SRPT), a biotechnology company specializing in precision genetic medicines for rare diseases with a market capitalization of $11.2 billion, stands at a critical juncture ...

Joshua Mercieca defied the odds after he was diagnosed with the muscle-wasting condition Duchenne muscular dystrophy at a ...